Enzyme Replacement Therapy

Enzyme replacement therapy (ERT) for MPS VI was approved by the FDA in 2005 and by the EMEA in 2006. This therapy provides patients with a recombinant version of the human enzyme N-acetylgalactosamine 4-sulfatase (arylsulfatase B, or ASB), which is deficient in patients with MPS VI. NAGLAZYME (galsulfase) for MPS VI treats the underlying enzyme deficiency by providing recombinant human ASB (rhASB) intravenously, which can be taken up by lysosomes and reduce GAG storage.

Targeting rhASB to the Lysosome: Proper glycosylation of rhASB—including the mannose 6-phosphate signal—is crucial to ensuring that the enzyme is absorbed by cells and trafficked to the proper intracellular compartment—the lysosome.

NAGLAZYME(galsulfase) is indicated for patients with mucopolysaccharidosis VI (MPS VI). NAGLAZYME has been shown to improve walking and stair-climbing capacity.

Important Safety Information

The most common adverse events observed in clinical trials in patients treated with NAGLAZYME were headache, fever, arthralgia, vomiting, upper respiratory infections, abdominal pain, diarrhea, ear pain, cough, and otitis media. Severe reactions included angioneurotic edema, hypotension, dyspnea, bronchospasm, respiratory distress, apnea, and urticaria. The most common symptoms of infusion reactions included fever, chills/rigors, headache, rash, and mild to moderate urticaria. Nausea, vomiting, elevated blood pressure, retrosternal pain, abdominal pain, malaise, and joint pain were also reported. No patients discontinued infusions of NAGLAZYME for adverse events and all patients who completed the double-blind portion of the trial continued to receive weekly infusions of NAGLAZYME. Nearly all patients developed antibodies as a result of treatment, but the level of the immune response did not correlate with the severity of adverse events. Because antihistamine use may increase the risk of apneic episodes, evaluation of airway patency should be considered prior to the initiation of treatment. Consideration to delay infusion of NAGLAZYME should be given when treating patients who present with an acute febrile or respiratory illness.
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